Oncology

HR+/HER2- Studies in Breast Cancer

  • Exploring ribociclib with novel/emergent compounds
  • Utilizing real-world data (RWD) and/or digital health technologies
  • Enhances the treatment experience of patients

Out of Scope

  • Any area outside HR+/HER2- breast cancer
  • Any study in overlap with ongoing Novartis-sponsored/supported studies

GEP & Bronchopulmonary NET

  • Re-treatment/Re-challenge with 177LuDOTATATE (after initial 4 cycles)
  • Combinations of 177LuDOTATATE with other agents with potential to improve efficacy
  • Sequencing studies with 177LuDOTATATE
  • Long-term safety of 177LuDOTATATE
  • Efficacy/Safety of 177LuDOTATATE in specific patient subgroups

Other SSTR+ Tumors

  • Role of 177LuDOTATATE in the management of patients with other SSTR+ tumors

NETSPOT for Imaging

  • Role of 68GaDOTATATE in GEP-NET and other SSTR2+ tumors

Imaging Studies in FAP-expressing Solid Tumors

  • Role of FAP PET in diagnosis, staging, clinical decision-making, and treatment response
  • Studies exploring FAP PET as an imaging biomarker: correlation with other biomarkers such as histological, molecular, and genetic subtypes
  • Understanding FAP expression in benign/inflammatory processes in relation to FAP RLT safety and patient selection for therapy
  • Use of PSMA-targeted PET imaging agents in prostate cancer (e.g., patient selection, treatment assessment)
  • Treatments up-regulating PSMA expression in prostate cancer
  • Methods of preventing and/or managing RLT-induced xerostomia
  • Real-world evidence in prostate cancer for 225Ac-PSMA-617
  • Health disparities in advanced prostate cancer
  • Sequencing with 177Lu-PSMA-617
  • Essential factors for selecting patients for tisagenlecleucel therapy to improve safety and/or response for their approved indications
  • Essential factors for sequencing tisagenlecleucel therapy with other therapies and determining outcomes for their approved indications
  • Novel combinations of therapies with tisagenlecleucel to improve response and/or safety for their approved indications
  • Study outcomes of tisagenlecleucel administered at various sites (e.g., inpatient, outpatient, community hospital, community practice) for their approved indications

CML-CP in Earlier Lines (1L & 2L)

  • Sequencing of TKIs, clinical efficacy, and safety in real-world settings
  • Patient-reported outcomes (PROs) and quality-of-life issues with current CML therapies
  • Biomarkers for TFR and safety
  • Long-term safety and tolerability
  • Studies aiming to improve eligibility to attempt TFR and reduce the risk of relapse after TFR attempts
  • Response to asciminib in patients with pre-existing BCR::ABL1 mutations other than T315I, or treatment approaches in patients with emerging mutations under asciminib, including compound mutations

CML-BC and Ph+ ALL

  • Efficacy and safety of asciminib in selected ALL settings (Ph+, Ph-like)
  • Exploratory high-risk advanced phase CML populations such as patients with additional genomic alterations
  • TKI-based combinations addressing high unmet need populations (CML-AP/BC)

Out of Scope

  • Use in non-BCR::ABL diseases
  • Investigating retreatment or extended treatment with 177Lu-PSMA-617
  • Real-world evidence in prostate cancer for 177Lu-PSMA-617
  • Health disparities in advanced prostate cancer
CRM
  • ASCVD MOA – atherosclerotic plaque composition changes
  • Differentiation of inclisiran vs. other LLTs in a real-world setting (e.g., adherence, implementation)

Non-drug IITs

Epidemiology associated with elevated Lp(a)

  • Patient characterization, identification, and genetic risk across sub-groups.
  • Association and impact on different types of CVD (e.g., ischemic stroke, PAD), polyvascular disease, and other cardiovascular-related diseases.

Distinct and unique pathophysiology of Lp(a)-related CVD

  • Insights on the pro-thrombotic mechanisms impacted by Lp(a).
  • Unique features of Lp(a).

Patient perception of the contribution of Lp(a) to CVD and cardiovascular risk

Lp(a) testing and global cardiovascular risk management

  • Implementation of Lp(a) testing in CVD risk evaluation.
  • Clinical and economic value of Lp(a) testing.

Out of Scope:

  • Relationships between Lp(a) and LDL-C, and their independent contributions to Lp(a)-related disease risk.
  • Lp(a) in non-cardiovascular-related disease.

Disease-related

  • Studies evaluating the role of the endothelin pathway in proteinuric kidney diseases
  • Patient journey mapping and practice change initiatives to improve outcomes and reduce disparities

Drug-related

  • Studies on combination strategies with atrasentan in IgAN

Out of Scope:

  • Pediatric studies involving drug treatment
  • Studies exploring dosing regimens other than the currently FDA-approved dose
  • Head-to-head comparisons with other approved products
  • Studies including patients with eGFR <15 mL/min/1.73 m2 (CKD stage 5)

Disease-related

Studies evaluating the role of the complement system in complement-mediated kidney diseases.

Patient journey mapping and practice change initiatives to improve outcomes and reduce disparities.

Drug-related

Studies on combination strategies with iptacopan in IgAN.

Out of Scope

Pediatric studies (with drug).

Head-to-head comparisons with other approved products.

Studies including patients with GFR <20 mL/min/1.73 m2.

With drug

  • Mechanistic studies in PNH.
  • Studies evaluating factors associated with or predictive of treatment outcome in PNH.

Without drug

  • Approaches to facilitating and expediting diagnosis
  • Identification of biomarkers that lead to better characterization, management, or correlation with outcomes.

Out of Scope

  • Pediatric studies.
  • Clinical trials exploring different dosing regimens than those currently being evaluated in the clinical development program.
  • Clinical trials combining iptacopan with immunosuppressant and anti-C5 treatments.
  • Head-to-head comparisons.
  • Studies in other non-complement-mediated hematologic diseases.
Neuroscience

Multiple Sclerosis

  • The experience of use of OMB in sub-populations of RMS (e.g., African American and Hispanic patients, and different age groups).
  • The impact of OMB on MS comorbidities and patient-centric outcomes.
  • The therapeutic role of OMB in MS, including efficacy, safety, tolerability, and use in treatment-naive patients.
  • The impact of OMB on both fluid and digital biomarkers in MS.
  • The pathophysiology of MS (including the mechanism of action of OMB and its effects on MS pathophysiology) and the burden of disease associated with MS (including the impact of OMB).
  • Innovative neuroimaging techniques used to measure biomarkers of MS disease, MS inflammation, axonal integrity, and function (including the effects of OMB).
  • The long-term impact of B-cell therapy on the immune system.
  • Impact of B-cell-depleting therapies on immune system function over time, with particular focus on the non-B-cell compartment.

Multiple Sclerosis

  • The role for remibrutinib in sequencing of treatments.
  • The impact on imaging, including SELs, PRLs, and cortical lesions.
  • The impact on the biology of progression, including PET imaging, cognition, fatigue, and depression outcomes.
  • Effect of remibrutinib on CNS processes, including blood–brain barrier (BBB) transmigration and microglial activation.
  • The proteome profiling effects of remibrutinib.

Myasthenia Gravis

  • Development of biomarkers and endpoint exploration for clinical trial use.
  • Impact on macrophage involvement in disease pathophysiology at the neuromuscular junction (NMJ).
  • Effect of remibrutinib on steroid use and its potential to reduce corticosteroid exposure.

In Scope

  • Holistic and societal impact of Zolgensma IV, including clinical transformation, economic impact, quality of life, and societal effects.
  • Biomarkers for efficacy.

Out of Scope

  • Clinical trials involving Zolgensma IV re-dosing.
  • Studies of alternative Zolgensma IV doses or maximum dose.
  • Basic science research requesting the use of Zolgensma IV.
  • Methods or processes to assess the efficacy and durability of Zolgensma IV (e.g., bulbar function).

In Scope

  • Long-term, multi-domain functional and clinical outcomes, including sleep, bulbar function, head control, scoliosis, respiratory function, and overall independence.
  • Reproductive outcomes, including fertility and reproductive health.
  • Biomarker and mechanistic evidence generation, including biomarkers predictive of clinical and functional response and durability.
  • Overall survival, caregiver and societal impact, long-term care needs and cost/resource implications, and the impact of delaying treatment.

Out of Scope

  • Interventional studies of OAV101 IT in patient types not included in clinical trials.
  • Clinical trials involving OAV101 IT re-dosing or OAV101 IT dosing following OAV101 IV.
  • Studies of alternative OAV101 IT doses or maximum dose.
  • Head-to-head comparisons with other therapies and combination with other MDTs.
  • Studies of OAV101 IT in patients under 2 years of age.
  • Comparative studies between Zolgensma IV and OAV101 IT.
Immunology

Disease Related Research

  • Population-based epidemiology studies; studies investigating the impact of urticaria on patients; studies of real-world treatment patterns (including, for example, overuse of corticosteroids and impact on sleep); and studies of patient preference, patient experience, and satisfaction (qualitative).
  • Studies employing digital technology, such as in silico models, AI-enabled or machine learning techniques, telemedicine, and related approaches to predict disease trajectories, treatment response, disease modification, and similar outcomes.
  • Studies investigating innovative tools to support urticaria management, including digital applications and sleep-related tools.
  • Long-term CSU/CIndU observational studies and secondary use of data.

Clinical Studies

  • Studies with remibrutinib in CSU/CIndU.
  • Proof-of-concept studies in new allergic and dermatologic indications.

Mechanistic Studies

  • Mechanistic studies assessing the effects of remibrutinib on mast cells, basophils, B cells, and other relevant immune pathways in vitro or ex vivo.

Out of Scope

  • Head-to-head comparisons of remibrutinib with other active treatments.
  • Studies investigating remibrutinib in combination with biologics.
  • Alternative dosing regimens to the 25 mg b.i.d. regimen developed in the CSU Phase 3 clinical program.
  • Sjogren’s US epidemiology and systemic disease characterization.
  • Sjogren's classification, systemic disease recognition, and clinical assessment.
  • Sjogren's disease progression: use of ultrasound, clinical assessments, and/or biomarkers.
  • Sjogren’s symptoms: evidence generation using existing PROs or new SjD symptom assessment modalities.
  • Sjogren's disease organ domains: generation of evidence in key disease domains.
  • Sjogren's disease and concomitant conditions (e.g., rheumatoid arthritis, lupus) and associated outcomes.
  • Sjogren's disease in subpopulations (e.g., African American, Hispanic) and associated outcomes.
  • Sjogren's disease burden: clinical, social, economic, and humanistic aspects.
  • Biomarker-informed assessment and monitoring in SjD.
  • SjD serologic and immunologic characterization and association with clinical outcomes.
  • Tissue-level immune activity and disease progression in SjD.